Back to Trials
NCT05371613PHASE2, PHASE3Recruiting

A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Denali Therapeutics Inc.

Start Date

7/21/2022

Completion Date

12/1/2027

Summary

This is a Phase 2/3, multiregional, two-arm, double-blind, randomized, active (standard-of-care)-controlled study of the efficacy and safety of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme-replacement therapy (ERT) for mucopolysaccharidosis type II (MPS II). Participants may also qualify to enter an open-label treatment phase with DNL310 or idursulfase based on pre-specified criteria.

Eligibility Criteria

Age Range: 2 years to 25 years

Key Inclusion Criteria: * Participants aged ≥2 to \<6 years (Cohort A) or ≥6 to \<26 years (Cohort B) * Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS II) * Have no history of treatment with enzyme replacement therapy (ERT) OR not have received continuous ERT for 4 months prior to screening OR be on maintenance ERT and have tolerated idursulfase for a minimum of 4 months prior to screening Key Exclusion Criteria: * Have a documented mutation of other genes or genetic diagnosis accounting for developmental delay * Previously received an iduronate 2-sulfatase (IDS) gene therapy or stem cell therapy * Received any CNS-targeted MPS ERT within 6 months prior to screening * Have a contraindication for lumbar punctures and/or magnetic resonance imaging (MRI) * Participated in any other investigational drug study or used an investigational drug within 60 days prior to screening or intend to receive another investigational drug during the study

Interventions

DRUG

tividenofusp alfa

DRUG

idursulfase

Interested in This Trial?

Contact the trial locations directly using the information below to learn more about enrollment.

Expressing interest lets you review the study and consent before we connect you with the research site.

Conditions

Mucopolysaccharidosis II

Locations

UCSF Benioff Children's Hospital Oakland

Oakland, California 94609

United States

Ann and Robert H Lurie Children's Hospital of Chicago

Chicago, Illinois 60611

United States

Hackensack University Medical Center

Hackensack, New Jersey 07601

United States

UNC Children's Research Institute

Chapel Hill, North Carolina 27514

United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio 45229

United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania 19104

United States

The University of Texas Medical School at Houston

Houston, Texas 77030

United States

University of Utah, PPDS

Salt Lake City, Utah 84132

United States

Sanatorio Mater Dei

Buenos Aires,

Argentina

Women's and Children's Hospital

North Adelaide,

Australia

UZ Antwerpen

Antwerp, Antwerpen 2650

Belgium

Universitair Ziekenhuis Brussel

Jette, Brussels Capital 1090

Belgium

Hospital de Clínicas de Porto Alegre (HCPA) - PPDS

Porto Alegre,

Brazil

Instituto Fernandes Figueira

Rio de Janeiro,

Brazil

University of Alberta - Faculty of Medicine & Dentistry

Edmonton, Alberta

Canada

Hospital for Sick Children

Toronto, Ontario M5G1X8

Canada

McGill University Health Center

Montreal, Quebec H4A3J1

Canada

Vseobecna Fakultni Nemocnice V Praze

Prague, 128 08

Czechia

Hôpital Jeanne de Flandre

Lille, 59000

France

SphinCS

Höchheim, Hochheim 65239

Germany

Medizinische Universität Lausitz - Carl Thiem

Cottbus,

Germany

Azienda Sanitaria Universitaria Friuli Centrale - PO Universitario Santa Maria della Misericordia

Udine, 33100

Italy

Erasmus Medical Center - Sophia Children's Hospital

Rotterdam, Rotterdam 3000

Netherlands

Hospital Universitario Vall d'Hebron

Barcelona, Barcelona 08035

Spain

Hospital Infantil Universitario Niño Jesus

Madrid, Madrid 28009

Spain

Drottning Silvias Barn Och Ungdomssjukhus

Gothenburg, 416 85

Sweden

Cukurova University Medical Faculty Balcali Hospital

Adana, 1330

Turkey (Türkiye)

Gazi Universitesi Tip Fakultesi

Çankaya, 06500

Turkey (Türkiye)

Great Ormond Street Hospital for Children

London, London WC1N 3JH

United Kingdom

Birmingham Women's and Children's NHS Foundation Trust

Birmingham,

United Kingdom

Royal Free Hospital

London, NW3 2QG

United Kingdom

Salford Royal Hospital

Salford, M6 8HD

United Kingdom